Vimizim

Generic Name: elosulfase alfa
Date of Approval: February 14, 2014
Company: BioMarin Pharmaceutical Inc.

Treatment for: Mucopolysaccharidosis Type IVA

FDA Approves Vimizim

The U.S. Food and Drug Administration (FDA) has approved Vimizim (elosulfase alfa), an enzyme replacement therapy for patients with Mucopolysaccharidosis type IVA or MPS IVA, also called Morquio A syndrome. Mucopolysaccharidosis type IVA is a rare, autosomal recessive lysosomal storage disease caused by a deficiency in N-acetylgalactosamine-6-sulfate sulfatase (GALNS) which is an enzyme involved in glycosaminoglycan (GAG) metabolism.

Incompletely broken down glycosaminoglycans remain stored in cells in the body and excessive storage causes problems with with bone development, growth and mobility. Other symptoms may include hearing loss, corneal clouding, and heart disease.

What is Vimizim?

Vimizim is an enzyme replacement therapy used to treat a rare congenital enzyme disorder called mucopolysaccharidosis IVA, also called Morquio A syndrome.

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Mucopolysaccharidosis IVA is a metabolic disorder which can lead to problems with bone development, growth and mobility.

Vimizim may improve symptoms in people with this condition, however it is not a cure.

Important information

Life-threatening allergic reactions, known as anaphylaxis, can occur during Vimizim (elosulfase alfa) infusions. Typical signs of anaphylaxis include cough, rash, throat tightness, hives, flushing, changes in skin color, low blood pressure, shortness of breath, chest pain, and gastrointestinal symptoms such as nausea, abdominal pain, retching, and vomiting. You should always have medical support readily available during the time the infusion is administered, and for an appropriate period following administration.

Hypersensitivity reactions can occur up to six days after infusion. Frequent symptoms of hypersensitivity reactions include anaphylactic reactions, itching, swelling, cough, shortness of breath, and flushing.

Antihistamines should be administered prior to infusion because of the potential for hypersensitivity reactions.

If severe hypersensitivity reactions occur, the infusion should be stopped immediately and appropriate treatment should be initiated. You are more likely to experience life-threatening complications from hypersensitivity reactions if you have a fever or respiratory illness at the time of administration.

Before receiving this medicine

You should not use this medication if you are allergic to elosulfase alfa.

To make sure Vimizim is safe for you, tell your doctor if you have:

  • asthma, or sleep apnea (breathing stops during sleep); or
  • fever, flu symptoms, or a common cold.

FDA Pregnancy Category C. It is not known whether Vimizim will harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant while using this medication. There is a Morquio A Registry that collects data on pregnant women with MPS IVA who are treated with Vimizim. Call 1-800-983-4587 for information and enrollment.

See also: Pregnancy and breastfeeding warnings (in more detail)

It is not known whether Vimizim is present in human milk or if it could harm a nursing baby. Tell your doctor if you are breast-feeding a baby. There is a Morquio A Registry that also collects data on breastfeeding women with MPS IVA who are treated with Vimizim. Call 1-800-983-4587 for information and enrollment.

How is Vimizim given?

Vimizim is injected slowly into a vein through an IV infusion. You will most likely receive this injection in a clinic or hospital setting.

The infusion can take between 3.5 to 4.5 hours, and is given once every week.

Your doctor may also prescribe other medications to help prevent an allergic reaction to Vimizim. Take all of your medications as directed.

Your doctor will need to check your progress while you are using Vimizim.

What happens if I miss a dose?

Call your doctor for instructions if you miss an appointment for your Vimizim injection.

What happens if I overdose?

Since this medication is given by a healthcare professional in a medical setting, an overdose is unlikely to occur.

What should I avoid?

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

Vimizim side effects

Some side effects may occur during the Vimizim infusion, or up to six days afterward. Tell your caregiver right away if you feel dizzy, light-headed, itchy, or if you have hives, chest pain, stomach pain, fever, trouble breathing, eye irritation, or swelling in your face.

Get emergency medical help if you have any of these signs of an allergic reaction: hives; difficulty breathing; feeling like you might pass out; swelling of your face, lips, tongue, or throat.

Even though it may not be a side effect of Vimizim, increased pressure on the spinal cord is a complication of MPS IVA that may occur while you are using Vimizim. Tell your doctor right away if you have any symptoms of spinal cord compression: back pain, loss of movement in any part of your body, loss of bowel or bladder control.

Call your doctor at once if you have:

  • snoring or sleep apnea, trouble breathing;
  • shortness of breath (even with mild exertion);
  • swelling, rapid weight gain;
  • eye redness; or
  • fever, chills, flu symptoms, pale skin, easy bruising or bleeding, unusual weakness.

Common side effects may include:

  • pain, fever;
  • mild rash or itching;
  • headache, ear pain, joint pain; or
  • nausea, vomiting, stomach pain.

This is not a complete list of side effects and others may occur. Tell your doctor about any unusual or bothersome side effect. You may report side effects to FDA at 1-800-FDA-1088.

See also: Side effects (in more detail)

Vimizim dosing information

Usual Adult Dose for Mucopolysaccharidosis Type IVA:

2 mg/kg body weight administered once every week as an intravenous infusion.

Pretreatment with antihistamines with or without antipyretics is recommended 30 to 60 minutes prior to the start of the infusion.

Usual Pediatric Dose for Mucopolysaccharidosis Type IVA:

5 years or older: 2 mg/kg body weight administered once every week as an intravenous infusion.

Pretreatment with antihistamines with or without antipyretics is recommended 30 to 60 minutes prior to the start of the infusion.

What other drugs will affect this medicine?

Other drugs may interact with Vimizim, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell each of your health care providers about all medicines you use now and any medicine you start or stop using.

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